Rita Faria’s journal round-up for 4th November 2019

Every Monday our authors provide a round-up of some of the most recently published peer reviewed articles from the field. We don’t cover everything, or even what’s most important – just a few papers that have interested the author. Visit our Resources page for links to more journals or follow the HealthEconBot. If you’d like to write one of our weekly journal round-ups, get in touch.

The marginal benefits of healthcare spending in the Netherlands: estimating cost-effectiveness thresholds using a translog production function. Health Economics [PubMed] Published 30th August 2019

The marginal productivity of the healthcare sector or, as commonly known, the supply-side cost-effectiveness threshold, is a hot topic right now. A few years ago, we could only guess at the magnitude of health that was displaced by reimbursing expensive and not-that-beneficial drugs. Since the seminal work by Karl Claxton and colleagues, we have started to have a pretty good idea of what we’re giving up.

This paper by Niek Stadhouders and colleagues adds to this literature by estimating the marginal productivity of hospital care in the Netherlands. Spoiler alert: they estimated that hospital care generates 1 QALY for around €74,000 at the margin, with 95% confidence intervals ranging from €53,000 to €94,000. Remarkably, it’s close to the Dutch upper reference value for the cost-effectiveness threshold at €80,000!

The approach for estimation is quite elaborate because it required building QALYs and costs, and accounting for the effect of mortality on costs. The diagram in Figure 1 is excellent in explaining it. Their approach is different from the Claxton et al method, in that they corrected for the cost due to changes in mortality directly rather than via an instrumental variable analysis. To estimate the marginal effect of spending on health, they use a translog function. The confidence intervals are generated with Monte Carlo simulation and various robustness checks are presented.

This is a fantastic paper, which will be sure to have important policy implications. Analysts conducting cost-effectiveness analysis in the Netherlands, do take note.

Mixed-effects models for health care longitudinal data with an informative visiting process: a Monte Carlo simulation study. Statistica Neerlandica Published 5th September 2019

Electronic health records are the current big thing in health economics research, but they’re not without challenges. One issue is that the data reflects the clinical management, rather than a trial protocol. This means that doctors may test more severe patients more often. For example, people with higher cholesterol may get more frequent cholesterol tests. The challenge is that traditional methods for longitudinal data assume independence between observation times and disease severity.

Alessandro Gasparini and colleagues set out to solve this problem. They propose using inverse intensity of visit weighting within a mixed-methods model framework. Importantly, they provide a Stata package that includes the method. It’s part of the wide ranging and super-useful merlin package.

It was great to see how the method works with the directed acyclic graph. Essentially, after controlling for confounders, the longitudinal outcome and the observation process are associated through shared random effects. By assuming a distribution for the shared random effects, the model blocks the path between the outcome and the observation process. It makes it sound easy!

The paper goes through the method, compares it with other methods in the literature in a simulation study, and applies to a real case study. It’s a brilliant paper that deserves a close look by all of those using electronic health records.

Alternative approaches for confounding adjustment in observational studies using weighting based on the propensity score: a primer for practitioners. BMJ [PubMed] Published 23rd October 2019

Would you like to use a propensity score method but don’t know where to start? Look no further! This paper by Rishi Desai and Jessica Franklin provides a practical guide to propensity score methods.

They start by explaining what a propensity score is and how it can be used, from matching to reweighting and regression adjustment. I particularly enjoyed reading about the importance of conceptualising the target of inference, that is, what treatment effect are we trying to estimate. In the medical literature, it is rare to see a paper that is clear on whether it is average treatment effect or average treatment effect among the treated population.

I found the algorithm for method selection really useful. Here, Rishi and Jessica describe the steps in the choice of the propensity score method and recommend their preferred method for each situation. The paper also includes the application of each method to the example of dabigatran versus warfarin for atrial fibrillation. Thanks to the graphs, we can visualise how the distribution of the propensity score changes for each method and depending on the target of inference.

This is an excellent paper to those starting their propensity score analyses, or for those who would like a refresher. It’s a keeper!

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Chris Sampson’s journal round-up for 12th August 2019

Every Monday our authors provide a round-up of some of the most recently published peer reviewed articles from the field. We don’t cover everything, or even what’s most important – just a few papers that have interested the author. Visit our Resources page for links to more journals or follow the HealthEconBot. If you’d like to write one of our weekly journal round-ups, get in touch.

Developing open-source models for the US health system: practical experiences and challenges to date with the Open-Source Value Project. PharmacoEconomics [PubMed] Published 7th August 2019

PharmacoEconomics will soon publish a themed issue on transparency in decision modelling (to which I’ve contributed), and this paper – I assume – is one that will feature. At least one output from the Open-Source Value Project has featured in these round-ups before. The purpose of this paper is to describe the experiences of the initiative in developing and releasing two open-source models, one in rheumatoid arthritis and one in lung cancer.

The authors outline the background to the project and its goal to develop credible models that are more tuned-in to stakeholders’ needs. By sharing the R and C++ source code, developing interactive web applications, and providing extensive documentation, the models are intended to be wholly transparent and flexible. The model development process also involves feedback from experts and the public, followed by revision and re-release. It’s a huge undertaking. The paper sets out the key challenges associated with this process, such as enabling stakeholders with different backgrounds to understand technical models and each other. The authors explain how they have addressed such difficulties along the way. The resource implications of this process are also challenging, because the time and expertise required are much greater than for run-of-the-mill decision models. The advantages of the tools used by the project, such as R and GitHub, are explained, and the paper provides some ammunition for the open-source movement. One of the best parts of the paper is the authors’ challenge to those who question open-source modelling on the basis of intellectual property concerns. For example, they state that, “Claiming intellectually property on the implementation of a relatively common modeling approach in Excel or other programming software, such as a partitioned survival model in oncology, seems a bit pointless.” Agreed.

The response to date from the community has been broadly positive, though there has been a lack of engagement from US decision-makers. Despite this, the initiative has managed to secure adequate funding. This paper is a valuable read for anyone involved in open-source modelling or in establishing a collaborative platform for the creation and dissemination of research tools.

Incorporating affordability concerns within cost-effectiveness analysis for health technology assessment. Value in Health Published 30th July 2019

The issue of affordability is proving to be a hard nut to crack for health economists. That’s probably because we’ve spent a very long time conducting incremental cost-effectiveness analyses that pay little or no attention to the budget constraint. This paper sets out to define a framework that finally brings affordability into the fold.

The author sets up an example with a decision-maker that seeks to maximise population health with a fixed budget – read, HTA agency – and the motivating example is new medicines for hepatitis C. The core of the proposal is an alternative decision rule. Rather than simply comparing the incremental cost-effectiveness ratio (ICER) to a fixed threshold, it incorporates a threshold that is a function of the budget impact. At it’s most basic, a bigger budget impact (all else equal) means a greater opportunity cost and thus a lower threshold. The author suggests doing away with the ICER (which is almost impossible to work with) and instead using net health benefits. In this framework, whether or not net health benefit is greater than zero depends on the size of the budget impact at any given ICER. If we accept the core principle that budget impact should be incorporated into the decision rule, it raises two other issues – time and uncertainty – which are also addressed in the paper. The framework moves us beyond the current focus on net present value, which ignores the distribution of costs over time beyond simply discounting future expenditure. Instead, the opportunity cost ‘threshold’ depends on the budget impact in each time period. The description of the framework also addresses uncertainty in budget impact, which requires the estimation of opportunity costs in each iteration of a probabilistic analysis.

The paper is thorough in setting out the calculations needed to implement this framework. If you’re conducting an economic evaluation of a technology that could have a non-marginal (big) budget impact, you should tag this on to your analysis plan. Once researchers start producing these estimates, we’ll be able to understand how important these differences could be for resource allocation decision-making and determine whether the likes of NICE ought to incorporate it into their methods guide.

Did UberX reduce ambulance volume? Health Economics [PubMed] [RePEc] Published 24th June 2019

In London, you can probably – at most times of day – get an Uber quicker than you can get an ambulance. That isn’t necessarily a bad thing, as ambulances aren’t there to provide convenience. But it does raise an interesting question. Could the availability of super-fast, low-cost, low-effort taxi hailing reduce pressure on ambulance services? If so, we might anticipate the effect to be greatest where people have to actually pay for ambulances.

This study combines data on Uber market entry in the US, by state and city, with ambulance rates. Between Q1 2012 and Q4 2015, the proportion of the US population with access to Uber rose from 0% to almost 25%. The authors are also able to distinguish ‘lights and sirens’ ambulance rides from ‘no lights and sirens’ rides. A difference-in-differences model estimates the ambulance rate for a given city by quarter-year. The analysis suggests that there was a significant decline in ambulance rates in the years following Uber’s entry to the market, implying an average of 1.2 fewer ambulance trips per 1,000 population per quarter.

There are some questionable results in here, including the fact that a larger effect was found for the ‘lights and sirens’ ambulance rate, so it’s not entirely clear what’s going on. The authors describe a variety of robustness checks for our consideration. Unfortunately, the discussion of the results is lacking in detail and insight, so readers need to figure it out themselves. I’d be very interested to see a similar analysis in the UK. I suspect that I would be inclined to opt for an Uber over an ambulance in many cases. And I wouldn’t have the usual concern about Uber exploiting its drivers, as I dare say ambulance drivers aren’t treated much better.

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Chris Sampson’s journal round-up for 20th May 2019

Every Monday our authors provide a round-up of some of the most recently published peer reviewed articles from the field. We don’t cover everything, or even what’s most important – just a few papers that have interested the author. Visit our Resources page for links to more journals or follow the HealthEconBot. If you’d like to write one of our weekly journal round-ups, get in touch.

A new method to determine the optimal willingness to pay in cost-effectiveness analysis. Value in Health Published 17th May 2019

Efforts to identify a robust estimate of the willingness to pay for a QALY have floundered. Mostly, these efforts have relied on asking people about their willingness to pay. In the UK, we have moved away from using such estimates as a basis for setting cost-effectiveness thresholds in the context of resource allocation decisions. Instead, we have attempted to identify the opportunity cost of a QALY, which is perhaps even more difficult, but more easy to justify in the context of a fixed budget. This paper seeks to inject new life into the willingness-to-pay approach by developing a method based on relative risk aversion.

The author outlines the relationship between relative risk aversion and the rate at which willingness-to-pay changes with income. Various candidate utility functions are described with respect to risk preferences, with a Weibull function being adopted for this framework. Estimates of relative risk aversion have been derived from numerous data sources, including labour supply, lottery experiments, and happiness surveys. These estimates from the literature are used to demonstrate the relationship between relative risk aversion and the ‘optimal’ willingness to pay (K), calibrated using the Weibull utility function. For an individual with ‘representative’ parameters plugged into their utility function, K is around twice the income level. K always increases with relative risk aversion.

Various normative questions are raised, including whether a uniform K should be adopted for everybody within the population, and whether individuals should be able to spend on health care on top of public provision. This approach certainly appears to be more straightforward than other approaches to estimating willingness-to-pay in health care, and may be well-suited to decentralised (US-style) resource allocation decision-making. It’s difficult to see how this framework could gain traction in the UK, but it’s good to see alternative approaches being proposed and I hope to see this work developed further.

Striving for a societal perspective: a framework for economic evaluations when costs and effects fall on multiple sectors and decision makers. Applied Health Economics and Health Policy [PubMed] Published 16th May 2019

I’ve always been sceptical of a ‘societal perspective’ in economic evaluation, and I have written in favour of a limited health care perspective. This is mostly for practical reasons. Being sufficiently exhaustive to identify a truly ‘societal’ perspective is so difficult that, in attempting to do so, there is a very high chance that you will produce estimates that are so inaccurate and imprecise that they are more dangerous than useful. But the fact is that there is no single decision-maker when it comes to public expenditure. Governments are made up of various departments, within which there are many levels and divisions. Not everybody will care about the health care perspective, so other objectives ought to be taken into account.

The purpose of this paper is to build on the idea of the ‘impact inventory’, described by the Second Panel on Cost-Effectiveness in Health and Medicine, which sought to address the challenge of multiple objectives. The extended framework described in this paper captures effects and opportunity costs associated with an intervention within various dimensions. These dimensions could (or should) align with decision-makers’ objectives. Trade-offs invariably require aggregation, and this aggregation could take place either within individuals or within dimensions – something not addressed by the Second Panel. The authors describe the implications of each approach to aggregation, providing visual representations of the impact inventory in each case. Aggregating within individuals requires a normative judgement about how each dimension is valued to the individual and then a judgement about how to aggregate for overall population net benefit. Aggregating across individuals within dimensions requires similar normative judgements. Where the chosen aggregation functions are linear and additive, both approaches will give the same results. But as soon as we start to consider equity concerns or more complex aggregation, we’ll see different decisions being indicated.

The authors adopt an example used by the Second Panel to demonstrate the decisions that would be made within a health-only perspective and then decisions that consider other dimensions. There could be a simple extension beyond health, such as including the impact on individuals’ consumption of other goods. Or it could be more complex, incorporating multiple dimensions, sectors, and decision-makers. For the more complex situation, the authors consider the inclusion of the criminal justice sector, introducing the number of crimes averted as an object of value.

It’s useful to think about the limitations of the Second Panel’s framing of the impact inventory and to make explicit the normative judgements involved. What this paper seems to be saying is that cross-sector decision-making is too complex to be adequately addressed by the Second Panel’s impact inventory. The framework described in this paper may be too abstract to be practically useful, and too vague to be foundational. But the complexities and challenges in multi-sector economic evaluation need to be spelt out – there is no simple solution.

Advanced data visualisation in health economics and outcomes research: opportunities and challenges. Applied Health Economics and Health Policy [PubMed] Published 4th May 2019

Computers can make your research findings look cool, which can help make people pay attention. But data visualisation can also be used as part of the research process and provide a means of more intuitively (and accurately) communicating research findings. The data sets used by health economists are getting bigger, which provides more opportunity and need for effective visualisation. The authors of this paper suggest that data visualisation techniques could be more widely adopted in our field, but that there are challenges and potential pitfalls to consider.

Decision modelling is an obvious context in which to use data visualisation, because models tend to involve large numbers of simulations. Dynamic visualisations can provide a means by which to better understand what is going on in these simulations, particularly with respect to uncertainty in estimates associated with alternative model structures or parameters. If paired with interactive models and customised dashboards, visualisation can make complex models accessible to non-expert users. Communicating patient outcomes data is also highlighted as a potential application, aiding the characterisation of differences between groups of individuals and alternative outcome measures.

Yet, there are barriers to wider use of visualisation. There is some scepticism about bias in underlying analyses, and end users don’t want to be bamboozled by snazzy graphics. The fact that journal articles are still the primary mode of communicating research findings is a problem, as you can’t have dynamic visualisations in a PDF. There’s also a learning curve for analysts wishing to develop complex visualisations. Hopefully, opportunities will be identified for two-way learning between the health economics world and data scientists more accustomed to data visualisation.

The authors provide several examples (static in the publication, but with links to live tools), to demonstrate the types of visualisations that can be created. Generally speaking, complex visualisations are proposed as complements to our traditional presentations of results, such as cost-effectiveness acceptability curves, rather than as alternatives. The key thing is to maintain credibility by ensuring that data visualisation is used to describe data in a more accurate and meaningful way, and to avoid exaggeration of research findings. It probably won’t be long until we see a set of good practice guidelines being developed for our field.

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